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Simply Stated: Understanding Myotonia Congenita
Myotonia congenita (MC) is a rare, inherited neuromuscular condition characterized by muscle stiffness (myotonia) present
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The unbearable weight of grief that smoothes the jagged edges
I type my columns on Tuesdays, but I write them in my head in the
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New analysis tracks Elevidys outcomes 3 years after treatment
Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys
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I define my story, even when Duchenne rewrites the script
I’ve often written about learning to live alongside uncertainty. Duchenne muscular dystrophy has a way
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Still playing my tune with a little help from my friends
In my previous column, I shared my concern about losing more strength due to the
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PPMD Joins Collaborative Research Effort to Decode How Human Muscle Regenerates
PPMD is proud to partner with the Muscular Dystrophy Association (MDA), the FSHD Society, and
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Making the Impossible Possible: One Man’s Journey on the Camino de Santiago de Compostela
Kurt Aguilar and his wife, Cathryn Domrose, in Vigo, Spain, before starting on the Camino
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Advocates say 2026 will be ‘an exciting time’ for DMD community
For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous
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Sarepta reports positive 3-year data from the EMBARK trial: 4-7 year olds treated with their microdystrophin gene therapy, Elevidys, have significantly slower disease progression
Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years
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Sarepta Shares Topline Three-Year EMBARK Data for ELEVIDYS in Ambulatory Duchenne Patients
Sarepta Therapeutics, Inc. has announced positive topline three-year functional results from Part 1-treated patients in
